AANS Neurosurgeon | Volume 26, Number 2, 2017

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Unexplored Therapeutic Targets for ALS

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A new study presented at the 14th International Conference on Endothelin: Physiology, Pathophysiology and Therapeutics, researchers from Johns Hopkins University identified an unexplored avenue of treatment for ALS. Endothelin (ET)-1, a small protein produced by blood vessel cells and a powerful vessel constrictor, is also produced by astrocytes, cells in the brain that studies are revealing play many roles in health and disease. ALS progression is associated with the dysfunction of astrocytes, and earlier studies have shown that ET-1 influences a number of cellular pathways implicated in ALS progression. This new study investigated whether levels of ET and its receptor, ET-B, were altered in the regions where nerve cells die in ALS. The researchers examined ALS patient-derived tissue samples and cells and a mouse model of ALS using a range of gene expression and protein measurement techniques. They found a higher level of ET-1 in astrocytes and a higher level of the ET-B receptor in nerve cells in regions affected in ALS. They also found variations in the gene sequences for ET-1 in patients with an inherited form of ALS. According to researchers, the endothelin system may represent an unexplored and potentially significant target for therapeutic intervention. To read more about this study, click here.

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